Mouse studies suggest
inhibiting RIPK3 could help treat forms of Gaucher's disease with
neurological symptoms. Enzyme replacement therapy to restore glucocerebrosidase
(GBA;
GCase)
does not treat neurological symptoms of the disease caused by neuron loss. In
a genetic mouse model of Gaucher's disease with neurological symptoms,
compared with littermates without the disease, Ripk3, which is involved in programmed
necrosis, was upregulated in brains. In a mouse model of Gaucher's disease
induced by an irreversible GCase inhibitor, Ripk3 knockout extended
lifespan and improved motor coordination, and it decreased neuron loss
compared with wild-type Ripk3 expression. Next steps include
developing blood brain barrier-penetrant small molecule inhibitors of the
RIPK3 pathway.

SciBX 7(5);
doi:10.1038/scibx.2014.146
Published online Feb. 6, 2014

Provisional patent
application filed in the U.S.; available for licensing

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